CureDuchenne is pleased to see that the FDA will hold an Advisory Committee Meeting (AdCom) for PTC Therapeutics’s drug Ataluren. We are hopeful for a full and fair review which will prioritize the patient and physician voice and experience.
Rare diseases are complicated and regulators need to hear about the real world benefit that patients experience; clinician endpoints are not enough. We may not have the perfect drug or the perfect trial. But if we can slow the disease progression and improve quality of life for Duchenne patients, it is a big win for our community.
This is an opportunity for the FDA to utilize the tools they have and allow access to drugs by patients that need them most. And, this is an opportunity for the entire Duchenne community to support each other at this very important AdCom. Please join CureDuchenne on September 28 in Washington DC for the Ataluren AdCom
See PTC announcement below.
PTC Therapeutics Announces FDA Advisory Committee Meeting for Ataluren for the Treatment of Nonsense Mutation Dystrophinopathy
– September 28th is FDA’s Tentative Date for Advisory Committee Meeting –
SOUTH PLAINFIELD, N.J., June 6, 2017 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28, 2017 to review the new drug application (NDA) for ataluren (Translarna™). Ataluren is an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation dystrophinopathy, including nonsense Duchenne muscular dystrophy (nmDMD).
The Company’s NDA submission for ataluren was granted standard review by the FDA on March 6, 2017. The FDA set a Prescription Drug User Fee Act (PDUFA) goal date of October 24, 2017 for completion of its review of the ataluren NDA.
About Duchenne Muscular Dystrophy
Primarily affecting males, Duchenne muscular dystrophy (DMD) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with DMD can lose the ability to walk as early as age ten, followed by loss of the use of their arms. DMD patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and twenties. It is estimated that a nonsense mutation is the cause of DMD in approximately 13 percent of patients.
About ataluren (Translarna™)
Ataluren, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna, tradename of ataluren, is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Ataluren is an investigational new drug in the United States. The development of ataluren has been supported by grants from Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA’s Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.
About PTC Therapeutics
PTC is a global biopharmaceutical company focused on the discovery, development, and commercialization of novel medicines using our expertise in RNA biology. PTC’s internally discovered pipeline addresses multiple therapeutic areas, including rare disorders and oncology. PTC has discovered all of its compounds currently under development using its proprietary technologies. Since its founding nearly 20 years ago, PTC’ mission has focused on developing treatments to fundamentally change the lives of patients living with rare genetic disorders. The company was founded in 1998 and is headquartered in South Plainfield, New Jersey. For more information on the company, please visit our website www.ptcbio.com.
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